clervonafusp alfa

Clervonafusp alfa (VAL-1221) is an investigational recombinant fusion protein designed to target both cytosolic and lysosomal glycogen. It comprises the Fab portion of a recombinant humanized cell-penetrating antibody (3E10 IgG) fused to recombinant human acid alpha-glucosidase (rhGAA). The antibody fragment utilizes the nucleoside transporter ENT-2 to penetrate cells and cross the blood-brain barrier, while the rhGAA component enters lysosomes via mannose-6-phosphate receptors (M6PRs). This dual-targeting mechanism allows clervonafusp alfa to degrade both cytosolic and lysosomal glycogen, addressing the underlying pathology of glycogen storage diseases. Initially developed by Valerion Therapeutics and subsequently by Parasail, the drug has been investigated for late-onset Pompe disease (glycogen storage disease type II) and Lafora disease, a fatal progressive myoclonic epilepsy characterized by the accumulation of polyglucosans (Lafora bodies) in the brain.

Other names

3E10Fab-GAAFabGAAhumanized 3E10Fab-GAAhumanized3E10Fab-GAAhumanized-3E10Fab-GAA